Current research activity

The clinical investigators at Children’s Minnesota are recognized nationally and internationally for their excellent care and outcomes. Our investigators engage in a wide range of research activities. They develop and implement their own projects, collaborate with investigators at other hospitals and universities, participate in registries and clinical trial networks, and consult as experts.

Current institutional research grants

IRGP Large Grant Program –  Awards up to $150,000 for 2 years

The Internal Research Grant Large Grant Program (IRGP) is designed to attract novel, high-impact research or inquiry into issues that affect child or adolescent health. In addition, funded projects should hold the promise of developing into long-term research and evaluation projects that will attract larger grants from NIH or other federal, state, or private funding agencies.


Early detection of DICER-1 related cancers, KrisAnn Schultz, MD

Project Summary: Pleuropulmonary blastoma (PPB) is the most common lung cancer of childhood and a sentinel manifestation of DICER-1 syndrome – a genetic predisposition to multiple, mainly pediatric tumor types. Early detection is imperative to improving outcomes in children with PPB. When PPB is discovered in its earliest form, most affected children survive; however, when PPB is more advanced at the time of diagnosis, outcomes remain poor. There are no effective treatments for recurrent or refractory disease. We hypothesize that the unique molecular pathogenesis of these tumors can be exploited  to facilitate   early detection of  new  or recurrent PPB. This project focuses on the preliminary clinical validation of novel “liquid biopsy” assays for sensitive detection and quantification of tumor burden in PPB and other DICER-related  cancers.


IRGP Small Grant Program – Awards up to $25,000 for 18 months

The IRGP small grant program is designed to support data collection for pilot research and evaluation of smaller projects which can serve as a basis for pursuing larger awards.


  • Point-of-care ultrasonography for intussusception: a randomized noninferiority trial, Kelly Bergmann DO, MS
  • Risk factors for prolonged recovery among children and adolescents with concussion: a retrospective cohort study, Amy Linabery PhD, MS
  • Don’t ask, don’t know: assessing health disparities for LGBTQ in a pediatric emergency department, Brianna McMichael MPH
  • Outcomes of speech surgery in patients with velopharyngeal insufficiency: a pilot study, Brittany Roby MD
  • Does implicit racial bias impact pediatric cardiopulmonary resuscitation (CPR) performance in a hybrid simulation, Samreen Vora MD, MHAM


Other funding programs 

Frequently additional funds are provided to the Research Institute to support specific research areas.  The following studies were grants awarded to projects in 2018 with a focus on neonatal outcomes.

  • Structural racism and income inequality: associations with adverse birth outcomes, Alicen Spaulding PhD and Amanda Nichol, MPH
  • Nutritional status as a predictor of cleft palate repair outcomes, Siva Chinnaduraie MD, MPH

Externally funded research at Children’s Minnesota

A Phase 1/2 multi-center study evaluating the safety and efficacy of KTE-C19 in pediatric and adolescent subjects with Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (r/r ALL) (ZUMA-4)

PI: Michael Richards, MD – Cancer and Blood Disorders Research Program

Funder: Kite Pharma

Currently, there are no good treatment options available for patients with pediatric acute lymphoblastic leukemia who never go into remission or who’s disease recurs after remission is achieved (r/r ALL). The most common subtype of leukemia is B-precursor ALL where the leukemia cells express a common molecule called CD19. Children’s is partnering with Kite Pharma on study to determine the safety and effectiveness of having the patient’s own healthy T-cells (a group of white blood cells) attack the CD-19 leukemia cells. In this process, a patient’s healthy T-cells are removed from their peripheral blood via a process called leukapheresis. The T-cells are sent to a laboratory where they are modified so that they recognize CD19-leukemia cells. When these new T-cells are re-introduced into the patient, they search out and attach themselves to the CD19-leukemia cells and kill them. These changed cells are called KTE-C19 cells (or CD19-targeting CAR-T). Several prior studies have shown that this type of therapy may be very effective treatment for r/r ALL in children and adolescents. Children’s is very excited to be able to offer this novel therapy to our patients.

NMTRC014 NMTT- Neuroblastoma Maintenance Therapy Trial using Difluoromethylornithine (DFMO)

PI: Jawhar Rawwas, MD – Cancer and Blood Disorders Research Program

Funder: KC Pharma LLC

As a member of the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC), Children’s is participating in a research study to evaluate the effectiveness a new therapy in preventing the relapse in neuroblastoma patients. Neuroblastoma is one of the most common tumors in children. Despite advances in therapy, outcomes for advanced stage neuroblastoma remain poor, and these children are at high risk of relapse following treatment. This clinical trial investigates the use of an oral medication called eflornithine (DFMO) administered daily following treatment of advanced stage neuroblastoma to determine the impact on relapse. Following promising results in lab studies, the NMTRC recently completed a trial using DFMO in pediatric patients. This trial compared outcomes of patients who underwent standard therapy, followed by two years of daily DFMO, to historical controls that finished the same standard therapy. While results are not yet published, initial indications merit further investigation into whether DFMO is an effective treatment for preventing relapse.

A Phase 3, rollover study to evaluate the safety of long-term treatment with Lumacaftor/Ivacaftor Combination Therapy in subjects aged 2 years and older with cystic fibrosis, Homozygous for the F508del-CFTR mutation 

PI: John McNamara, MD – Cystic Fibrosis Research Program

Funder: Vertex Pharmaceuticals

This is an open-label, rollover study being done to learn more about the long-term safety and effects from taking the combination of lumacaftor/ivacaftor in subjects aged 2 and older, who participated in a previous Vertex study (VX15-809-115 Part B). Lumacaftor/ivacaftor combination therapy has been approved for the treatment of CF in patients ages 6 year and older who are homozygous for the F508del mutation in the CFTR gene.  The F508del-CFTR mutation (most common) interferes with the ability of a CFTR protein and protein to reach and remain at the cell surface, as well as to open and close properly to transport chloride. The reduction or absence of chloride transport impairs fluid regulation (airway hydration) that causes accumulation of sticky mucus in the airways.

Preterm Epo Neuroprotection Trial (PENUT Trial)

PIs: Andrea Lampland, MD, Ellen Bendel-Stenzel, MD –  Neonatal Research Program

Funder: NINDS

Extremely low gestational age neonates are at high risk of death or neurodevelopmental impairment. Major morbidities, which include cerebral palsy, deafness, blindness, and mental disabilities are present in approximately 50% of surviving extremely preterm infants at school age. Long-term follow-up studies in these extremely premature infants are now increasingly identifying behavioral dysfunctions such as attention deficit disorder and autism spectrum disorder. The primary goal of this study is to determine whether administration of early high dose recombinant human erythropoietin (Epo) will improve the long term neurodevelopmental outcome of preterm infants <29 6/7 weeks of gestation will improve infants’ long term neurodevelo9pment outcomes measured at two years of age. The outcome of the trial would represent an important advance in the care of these neonates that could change the stand of care for high-risk infants, improving the lives of babies and their families, and decreasing the associated health care costs. During my neonatal fellowship training, I performed laboratory research examining various modes of ventilation and surfactant administration using animal models. As a staff neonatologist, I have been involved in enrollment for several research protocols involving multi-center randomized control clinical trials.

A Clinical Study to Evaluate the Safety and Effectiveness of the Infinity Acute Care System Workstation Neonatal Care Babylog VN500 Device in High Frequency Oscillatory Ventilation (HFOV) Mode in Very Low Birth Weight (VLBW) Neonates

PIs: Andrea Lampland, MD, Ellen Bendel-Stenzel, MD – Neonatal Research Program

Funder: Draeger Medical Systems

The Babylog VN500 with the HFOV mode has been available worldwide outside the US for the last few years. However, Draeger Medical Systems Inc. has over 15 years’ experience in HFOV. The purpose of this research study is to test the HFOV mode (a software option) on the ventilator to gain approval of the HFOV option also in the US. The study will compare results to retrospective data from similar babies treated with other FDA-approved HFOV devices during the last two years. This study will enroll babies born between 23 and 30 weeks gestation who require respiratory support from a ventilator. Significantly premature infants have a high risk of breathing difficulties because the lungs have not had a chance to fully develop and are not prepared to breathe by themselves. Often, premature babies of very low birth weight (VLBW) need help from a ventilator. A conventional ventilator delivers a normal volume of air in each breath, at a regular breath rate of about 30-60 breaths per minute. A high frequency oscillatory ventilator (HFOV) provides hundreds of tiny breaths per minute. HFOV devices are believed to be gentler on the lungs and make it easier for them to expand evenly. HFOV has been an established mode for premature babies for more than 20 years.

Education in Palliative and End-of-life Care Pediatrics

PI: Stefan Friedrichsdorf, MD – Pain Research Program

Funder: NIH

The Institute of Medicine and the National Cancer Policy Board have called for the integration of palliative care for all patients with cancer and other life-limiting diseases. In response, a number of programs have developed palliative care education, including the Education in Palliative and End-of-Life Care (EPEC) Project. And yet, in the U.S., the vast majority of infants, children, and teenagers with advanced illnesses who are near the end of life do not have access to interdisciplinary pediatric palliative care (PPC) services either in their community or at the nearest children’s hospital. In response to this deficiency, the aim of this research project is to, through a collaborative process, establish an Education in Palliative and End-of-life Care Pediatrics (EPEC-Pediatrics) Train-the-Trainer Curriculum. With knowledge gained through the EPEC-Pediatrics curriculum, physicians and Advanced Nurse Practitioners (APRN) will become ‘Trainers’ who will educate local interdisciplinary teams that provide care to children with cancer and hematological conditions, thus optimizing PPC outreach within the pediatric hematology/oncology context. The hypothesis is that a positive change will occur in EPEC-Pediatrics trained providers’ attitudes and knowledge and selected skills following completion of the training program.

Eliminating needle pain in children: A multisite program to reduce painful needle procedure experiences in the pediatric inpatient setting.

PI: Stefan Friedrichsdorf, MD – Pain Research Program

Funder: Mayday Fund

Since 2014 Children’s of Minnesota has initiated a system-wide program to facilitate inpatient needle procedure practice change (Toyota Lean Value Stream) through refinement and implementation of care delivery tools directed at reducing or eliminating needle pain in children: Expanding this quality improvement project, Childrenâ??s is partnering with four childrenâ??s hospitals in the US and Canada to implement and evaluate a standardized needle pain mediation protocol. Partner sites will participate in training to disseminate best practices in reducing pain associated with needle procedures. This project is aimed at creating a high quality tool kit that can be applied to various clinical environments utilizing best practices techniques for needle pain management in the pediatric inpatient setting.

HER-based Decision Support for Pediatric Acute Abdominal Pain in Emergency Care

PI: Anupam Kharbanda, MN – Emergency Department Research Program

Funder: NIH

Abdominal pain is one of the most common reasons for children and adolescents visits the pediatric Emergency Department (ED). Despite the common presentation, distinguishing appendicitis from other causes of abdominal pain remains difficult for ED physicians. CT scans have become increasingly common for diagnosing appendicitis. Increase in CT has not resulted in an increase in improved health outcomes for children with abdominal pain. Associated negative consequences of CT, including increased costs and exposure to radiation are concerning consequences of the increased rate of abdominal CT. The present study will develop and implement an electronic health record based decision support tool for clinicians in ED settings. The intervention will be evaluated at 14 hospitals in the Health Partners Care Network and the Kaiser Permanente Northern California Network. Specific aims for overall study are to design and implement a triage-based trigger to identify pediatric patients with acute abdominal pain at increased risk for appendicitis, to evaluate the impact of the full appy-CDS intervention on appropriate use of diagnostic imaging (CT and ultrasound) for pediatric patients with acute abdominal pain at risk for appendicitis, and to evaluate the impact of the full appy-CDS intervention on health care costs.

Glucagon-Like Peptide-1 Agonist Effects on Energy Balance in Hypothalamic Obesity

PI: Jennifer Abuzzahab, MD – Diabetes and Endocrinology Research

Funder: NIH

Excessive weight gain and its cardiometabolic sequela are frequent complications of hypothalamic tumors, a condition known as hypothalamic obesity (HO). Most tumors in this region are craniopharyngiomas (CP), which constitute 5-9% of childhood brain tumors. Patients with CP typically become obese and have more features of the metabolic syndrome compared to matched controls. Structural damage in several medial and posterior hypothalamic nuclei often lead to uncontrolled appetite, rapid weight gain, central insulin and leptin resistance, decreased sympathetic activity, low energy expenditure (EE), and increased energy storage i n adipose tissue. The weight gain is uncontrolled and not responsive to diet and exercise interventions. This clinical research study is being conducted for the drug subcutaneous drug Exenatide (also called ExQW or Bydureon) in suspension. This clinical research study’s primary objectives are to test changes in BMI, cardiometabolic factors, energy homeostasis, and determine predictors of glucagon-like peptide (GLP l ) treatment success in patients with hypothalamic obesity when given once weekly to obese patients with hypothalamic injury. Exenatide is approved for use in patients with diabetes but is experimental for patients with hypothalamic obesity.

Development of Novel Pediatric Diabetes Education Materials for Somali Immigrants

PI: Jennifer Kyllo, MD – Diabetes and Endocrinology Research

Funder: Child Health Collaborative Children with diabetes and their families go through diabetes education as part of their initial diagnosis in order to familiarize themselves with daily diabetes cares, including carbohydrates counting. However, patients within the Somali population may encounter difficulties during education due to language and cultural barriers, which can result in poorer diabetes control and knowledge compared to other populations. The University of Minnesota (UMN) and Children’s Minnesota (CHCMN) see a large number of Somali patients, with Children’s seeing roughly twice as many Somali patients as the University of Minnesota. Based on focus groups and a surveys obtained in a previous study with this population, there is a need to create more culturally accessible education materials in video and picture format to help improve diabetes knowledge and cares. The aim of this study is to develop an innovative, oral, video-based, patient-as-educator Type 1 Diabetes education program tailored to Somali immigrant families, taking into account such factors as parent literacy and numeracy level, culture, and religion. We will pilot the video education program on a different group of Somali T1D patients and parents followed at UMN and CHCM, who have previously received standard ADA T1D education and to assess improvement in diabetes knowledge as measured before, immediately after, and 3 months after participating in the program.

A multi-center, Phase 3, randomized, open-label, active-controlled, parallel group trial investigating the safety, tolerability, and efficacy of TransCon hGH administered once a week versus standard daily hGH replacement therapy over 52 weeks in prepubertal children with growth hormone deficiency (GHD)

PI: Jennifer Abuzzahab, MD – Diabetes and Endocrinology Research

Funder: Ascendis

Children participating in this trial have been diagnosed to have growth hormone deficiency. The standard treatment for growth hormone deficiency is daily injections of recombinant human growth hormone (rhGH). The purpose of this trial is to measure the efficacy and safety of once weekly injections of a new, longer-acting version of growth hormone (TransCon hGH) over a period of 1 year compared to daily growth hormone therapy.  TransCon hGH has the same active ingredient as the standard recombinant human growth hormone, but has been adapted to have a slower release and thus a longer action in the body, which makes it suitable for weekly (instead of daily) administration.